FDA grants orphan drug and fast-track designation to RLS-0071 for aGVHD
The FDA has granted orphan drug designation and fast track designation to RLS-0071 (ReAlta Life Sciences Inc) for the treatment of patients hospitalized for steroid-refractory acute graft-versus-host disease (aGVHD). The drug is an investigational drug based on ReAlta Life Sciences’ EPICC peptide platform and has the potential to modulate complement and innate inflammatory pathways. The drug is also currently in an open-label Phase 2 clinical trial for this patient population.1
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“We are very pleased to have received both Orphan Drug and Fast Track designation for RLS-0071 for the treatment of steroid-refractory acute graft-versus-host disease. This underscores the significant potential of RLS-0071 and its novel dual mechanism of action to address the critical unmet need of patients with this life-threatening condition,” said Kenji Cunnion, MD, MPH, Chief Medical Officer of ReAlta, in a press release. “RLS-0071 may address the limitations of current treatment options for patients with aGVHD. We remain committed to advancing our clinical development program in hopes of bringing this promising therapy to patients as quickly as possible.”1
In the Phase 2 trial, researchers included male and female patients aged 12 years and older who were hospitalized with steroid-refractory aGVHD after allogeneic hematopoietic stem cell transplantation (allo-HSCT). They also had an expected hospital length of stay of 1 week from the time of treatment initiation. Patients were excluded if they had had more than 1 allo-HSCT; current, previous, or planned systemic treatment with anything other than corticosteroids or ruxolitinib for aGVHD; an uncontrolled gastrointestinal infection; chronic GVHD; or had relapse of the underlying disease or were being treated for relapse after allo-HSCT.1
About the process
Title: Safety, PK, PD, dosing and efficacy of RLS-0071 for the treatment of hospitalized patients with steroid-refractory acute graft-versus-host disease (AURORA)
ClinicalTrials.gov ID: NCT06343792
Sponsor: ReAlta Life Sciences Inc
Completion date (estimated): September 2025
Cohorts included: 10 mg/kg every 8 hours for 7 days; 40 mg/kg every 8 hours for 7 days; 10 mg/kg every 8 hours for 14 days with concurrent ruxolitinib; 40 mg/kg every 8 hours for 14 days with concurrent ruxolitinib; 10 mg/kg for 7 days and then 10 mg/kg every 8 hours for 7 days with concurrent ruxolitinib; and 40 mg/kg every 8 hours for 7 days and then 40 mg/kg every 8 hours for 7 days with concurrent ruxolitinib.
The expansion cohort will be divided into two groups, each comprising 12 subjects, who will receive 10 or 40 mg/kg every 8 hours for 7 or 14 days in each cohort.2
Primary outcomes included the number of subjects with treatment-emergent adverse events (TEAEs) and serious TEAEs, and overall response rate. Secondary outcomes included the incidence of refractoriness, overall use of corticosteroids, initiation of additional/alternative treatments for aGVHD, change or shift in stage for lower gastrointestinal aGVHD, liver aGVHD, skin aGVHD, or upper gastrointestinal aGVHD from baseline based on MAGIC criteria, achievement of stage 0 or I for lower gastrointestinal aGVHD, liver aGVHD, skin aGVHD, or upper gastrointestinal aGVHD, change or shift in overall aGVHD grade, overall survival, relapse-free mortality, and length of hospital stay according to the clinical trial information.2
The company is also currently conducting two additional Phase 2 clinical trials with the drug, including for newborns with moderate or severe hypoxic-ischemic encephalopathy (NCT05778188) and for patients with acute exacerbations of chronic obstructive pulmonary disease (NCT06175065), the press release said.1
